{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,6,15]],"date-time":"2026-06-15T16:54:42Z","timestamp":1781542482440,"version":"3.54.5"},"reference-count":32,"publisher":"Wiley","issue":"2","license":[{"start":{"date-parts":[[2022,12,5]],"date-time":"2022-12-05T00:00:00Z","timestamp":1670198400000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Haemophilia"],"published-print":{"date-parts":[[2023,3]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Introduction<\/jats:title><jats:p>Current treatment for haemophilia A involves factor VIII replacement or non\u2010replacement (emicizumab) therapies, neither of which permanently normalise factor VIII levels. Gene therapy using adeno\u2010associated viral (AAV) vectors is an emerging long\u2010term treatment strategy for people with severe haemophilia A (PwSHA) that is likely to be available for clinical use in the near future.<\/jats:p><\/jats:sec><jats:sec><jats:title>Aim<\/jats:title><jats:p>This article proposes practical guidelines for the assessment, treatment, and follow\u2010up of potential PwSHA candidates for AAV\u2010based gene therapy.<\/jats:p><\/jats:sec><jats:sec><jats:title>Method<\/jats:title><jats:p>Using the Delphi method, a working group of Italian stakeholders with expertise in and knowledge of the care of adults with haemophilia A analysed literature for AAV\u2010based gene therapy and drafted a list of statements that were circulated to a panel of Italian peers. During two rounds of voting, panel members voted on their agreement with each statement to reach a consensus.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>The Delphi process yielded 40 statements regarding haemophilia A gene therapy, across five topics: (1) organisational model; (2) multidisciplinary team; (3) patient engagement; (4) laboratory surveillance; and (5) patient follow\u2010up and gene therapy outcomes. The consensus was reached for all 40 statements, with the second round of voting needed for five statements.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusion<\/jats:title><jats:p>Use of the hub\u2010and\u2010spoke organisational model and multidisciplinary teams are expected to optimise patient selection for gene therapy, as well as the management of dosing and patient follow\u2010up, patient engagement, laboratory surveillance, and patient expectations regarding outcomes. This approach should allow the benefits of AAV\u2010based gene therapy for haemophilia A to be maximised.<\/jats:p><\/jats:sec>","DOI":"10.1111\/hae.14709","type":"journal-article","created":{"date-parts":[[2022,12,5]],"date-time":"2022-12-05T21:25:21Z","timestamp":1670275521000},"page":"435-444","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":9,"title":["Laying the foundations for gene therapy in Italy for patients with haemophilia A: A Delphi consensus study"],"prefix":"10.1111","volume":"29","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-4973-1317","authenticated-orcid":false,"given":"Giancarlo","family":"Castaman","sequence":"first","affiliation":[{"name":"Center for Bleeding Disorders and Coagulation Department of Oncology Careggi University Hospital  Florence Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-0845-7940","authenticated-orcid":false,"given":"Christian","family":"Carulli","sequence":"additional","affiliation":[{"name":"Orthopaedic Clinic University of Florence  Florence Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-8066-8849","authenticated-orcid":false,"given":"Raimondo","family":"De Cristofaro","sequence":"additional","affiliation":[{"name":"Section of Haemorrhagic and Thrombotic Diseases Department of Diagnostic Imaging Oncological Radiotherapy and Haematology IRCCS A. Gemelli University Polyclinic Foundation  Rome Italy"},{"name":"Department of Medicine and Translational Surgery Sacred Heart University  Rome Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Marco","family":"Follino","sequence":"additional","affiliation":[{"name":"Fondazione Paracelso onlus  Milan Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Angelo","family":"Lupi","sequence":"additional","affiliation":[{"name":"Federation of Haemophilia Associations (FedEmo)  Milan Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-7113-4028","authenticated-orcid":false,"given":"Maria Elisa","family":"Mancuso","sequence":"additional","affiliation":[{"name":"Center for Thrombosis and Hemorrhagic Diseases IRCCS Humanitas Research Hospital  Rozzano Milan Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-8783-087X","authenticated-orcid":false,"given":"Maria Francesca","family":"Mansueto","sequence":"additional","affiliation":[{"name":"\u201cP. Giaccone\u201d University Hospital University of Palermo Haemophilia Centre and Haematology Unit  Palermo Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-8078-8402","authenticated-orcid":false,"given":"Angelo Claudio","family":"Molinari","sequence":"additional","affiliation":[{"name":"Regional Reference Centre for Haemorrhagic Diseases IRCCS Istituto Giannina Gaslini  Genoa Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Pietro","family":"Pasquetti","sequence":"additional","affiliation":[{"name":"Recovery and Rehabilitation Agency University Hospital of Careggi  Florence Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-7181-447X","authenticated-orcid":false,"given":"Cristina","family":"Santoro","sequence":"additional","affiliation":[{"name":"Division of Haematology Umberto I University Hospital  Rome Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-5081-8201","authenticated-orcid":false,"given":"Rita Carlotta","family":"Santoro","sequence":"additional","affiliation":[{"name":"Haemophilia, Haemostasis and Thrombosis Unit, Regional Reference Centre for Bleeding and Thrombosis Disorders Pugliese\u2010Ciaccio Hospital  Catanzaro Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-1641-6508","authenticated-orcid":false,"given":"Sergio","family":"Siragusa","sequence":"additional","affiliation":[{"name":"\u201cP. Giaccone\u201d University Hospital University of Palermo Haemophilia Centre and Haematology Unit  Palermo Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-4130-3601","authenticated-orcid":false,"given":"Luigi Piero","family":"Solimeno","sequence":"additional","affiliation":[{"name":"Division of Orthopaedic Surgery and Traumatology Foundation IRCCS Ca' Granda, Ospedale Maggiore Policlinico  Milan Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-1602-2776","authenticated-orcid":false,"given":"Armando","family":"Tripodi","sequence":"additional","affiliation":[{"name":"Angelo Bianchi Bonomi Haemophilia and Thrombosis Center Foundation IRCCS Ca\u2019 Granda Ospedale Maggiore Policlinico  Milan Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-6149-1155","authenticated-orcid":false,"given":"Ezio","family":"Zanon","sequence":"additional","affiliation":[{"name":"Haemophilia Centre Department of Medicine University Hospital of Padua  Padua Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-4235-7166","authenticated-orcid":false,"given":"Giovanni Di","family":"Minno","sequence":"additional","affiliation":[{"name":"Department of Clinical Medicine and Surgery \u201cFederico II University; 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